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Unlocking Transcription Factors to Address Unmet Medical Need

Developing small molecules with potential across multiple serious diseases

about us

Altay

Advancing Oral Transcription Factor Therapies for Patients with Limited Options

At Altay Therapeutics, we're unlocking the therapeutic potential of transcription factor inhibition to address serious, underserved diseases. Our pipeline includes the first oral small molecule inhibitor of DUX4 for facioscapulohumeral muscular dystrophy (FSHD) and a potential best-in-class STAT3 inhibitor with broad potential in cancer and chronic inflammatory diseases. We aim to bring novel transcription factor inhibitors to the clinic, not just to advance the science, but to offer differentiated therapeutic options to patients.

Altay

our science

Unlocking theTherapeutic Power of Transcription Factors

Transcription factors regulate the expression of hundreds of genes and play central roles in development, immunity and disease. Yet they've long been considered 'undruggable' due to their lack of defined binding pockets and complex structural dynamics.

At Altay, we've developed a therapeutic area-agnostic drug discovery platform that challenges this notion. By identifying novel allosteric sites - emerging only through dynamic structural changes in transcription factors - we design highly-selective small molecule inhibitors with the potential to be first- or best-in-class.

Pipeline

A Pipeline Rooted in Precision Biology

DX5057 DUX4 Inhibitor

FSHD

IND
ENABLING

PHASE 1/2A

2025

2026

STAT3 Inhibitor

Inflammatory
diseases & oncology

DEVELOPMENT
CANDIDATE

IND ENABLING

2026

Research & discovery Programs

ONCOLOGY

Lead Optimization

DX5057 for FSHD

Our lead program targets DUX4, the transcription factor that drives FSHD pathology. DX5057 is the first oral small molecule inhibitor of DUX4, showing preclinical efficacy comparable to the leading IV administered gene-targeting therapies, with the added benefit of ease of use and scalable delivery.

STAT3 Inhibitor for Inflammatory Diseases and Oncology

STAT3 is a validated transcription factor involved in immune dysregulation and cancer. Our inhibitor binds directly to the activated form of STAT3 and demonstrates nanomolar potency, broad therapeutic relevance and minimal off-target effects.

Undisclosed Program in Oncology

Our platform has also yielded early candidates against an undisclosed transcription factor with relevance in sarcomas and prostate cancer, continuing to expand the reach of our discovery engine.

Team

We are supported by a team of esteemed advisors and collaborators who bring a wealth of drug development expertise to Altay

Board of Directors
Team Members
Scientific Advisors

Investors & Funding Support